ISS, Noonan Syndrome, SGA, Turner Syndrome
Conditions
Brief summary
The purpose of this study is to find out if somapacitan is safe and how well somapacitan works in children either born small for gestational age or with Turner syndrome, Noonan syndrome or idiopathic short stature. Somapacitan is a new growth hormone medicine for treatment of low level of growth hormone. The study will last for about 3 years. During the study, the participants will be treated with somapacitan once a week. Somapacitan can be injected anytime during the day. The study doctor or nurse will show how to inject somapacitan, so that the participant knows how to do it at home.
Interventions
DRUGSomapacitan
Somapacitan 0.24 milligrams per kilograms per week (mg/kg/week) will be administered subcutaneously (s.c.) using PDS290 pen-injector.
Sponsors
Novo Nordisk A/S
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
10 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
Applicable to children with SGA: * Born small for gestational age (birth length below -2 SDS OR birth weight below -2 SDS OR both) (according to national standards). * Age: \- Male participants: Age equal to or above 11.0 years and below 18.0 years at screening. \- Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. * Open epiphyses; defined as bone age less than (\<) 14 years for females and bone age \< 16 years for males. * For Growth Hormone (GH) treatment naïve participants: Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention. Applicable to children with TS: • Diagnosis of TS according to local clinical practice. * Age: \- Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. * Open epiphyses; defined as bone age \< 14 years for females and bone age \< 16 years for males. * For GH treatment naïve participants: Impaired height defined as at least 2.0 standard deviation below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention. * For GH treatment naïve participants: Confirmed diagnosis of TS by 30-cell (or more) lymphocyte chromosomal analysis or confirmation of TS and TS mosaicism using comparative genomic hybridization (CGH)-array. Applicable to children with NS: * Diagnosis of NS according to local clinical practice. * Age: * Male participants: Age equal to or above 11.0 years and below 18.0 years at screening. * Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. * Open epiphyses; defined as bone age \< 14 years for females and bone age \< 16 years for males. * For GH treatment naïve participants: Clinical diagnosis of NS according to van der Burgt score list and genetic test result or confirmed mutation in any of the genes associated with NS before allocation. Applicable to children with ISS: * Age: \- Male participants: Age equal to or above 11.0 years and below 18.0 years at screening. \- Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. * Open epiphyses; defined as bone age \< 14 years for females and bone age \< 16 years for males. * For GH treatment naïve participants: Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening * For GH treatment naïve participants: Normal GH secretion (GH peak above 7 ng/mL) during GH stimulation test performed within 18 months prior to screening. * For GH treatment naïve participants: Bone age not delayed more than 2 years compared to chronological age at screening.
Exclusion criteria
* Children with suspected or confirmed growth hormone deficiency according to local practice. * Children diagnosed with diabetes mellitus or screening values from the central laboratory. * Fasting plasma glucose above or equal to 126 milligrams per deciliter (mg/dL) \[7.0 millimoles per litre (mmol/L)\] or * Glycated hemoglobin (HbA1c) above or equal to 6.5%. * Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening. * Children requiring inhaled glucocorticoid therapy at a dose greater than 400 micrograms per day (µg/day) of inhaled budesonide or equivalent (i.e., 250 µg/day for fluticasone propionate) for longer than 4 consecutive weeks within the last 12 months prior to screening. * History or known presence of malignancy including intracranial tumours. Applicable to children with SGA: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: * Poorly controlled or uncontrolled hormonal deficiencies. * Known chromosomal aneuploidy or significant gene mutations causing medical 'syndromes' with short stature, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, skeletal dysplasias, abnormal short stature homeobox (SHOX) gene analysis or absence of GH receptors. Applicable to children with TS: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: * Known family history of skeletal dysplasia. * Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. * Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease. * Mosaicism below 10%. * TS with Y-chromosome mosaicism where gonadectomy has not been performed. * New York Heart Association (NYHA) class II or above or requiring medication for any heart condition. Applicable to children with NS: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: * Known family history of skeletal dysplasia. * Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. * Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease. * Noonan-related disorders including but not limited to: Noonan syndrome with multiple lentigines (formerly called 'LEOPARD' syndrome), Noonan syndrome with loose anagen hair, cardiofaciocutaneous syndrome (CFC), Costello syndrome, neurofibromatosis type 1 (NF1) and Legius syndrome. Applicable to children with ISS: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: * Known family history of skeletal dysplasia. * Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. * Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease. * Poorly controlled or uncontrolled hormonal deficiencies. * Known chromosomal aneuploidy or significant gene mutations causing medical 'syndromes' with short stature, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, skeletal dysplasias, abnormal SHOX gene analysis or absence of GH receptors.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Adverse Events Reported for Turner Syndrome (TS)- Weeks 0 to 26 | From baseline (Week 0) to Week 26 | This outcome measure reported number of AEs in participants with short stature for indication TS. TS is a chromosomal disorder which leads to short stature. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Number of Adverse Events Reported for Noonan Syndrome- Weeks 0 to 26 | From baseline (Week 0) to Week 26 | This outcome measure reported number of AEs in participants with short stature for indication NS which is a genetically heterogeneous developmental disorder characterized by postnatally reduced growth and other major disorders. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Number of Adverse Events Reported for Idiopathic Short Stature (ISS)- Weeks 0 to 26 | From baseline (Week 0) to Week 26 | This outcome measure reported number of AEs in participants with short stature for indication ISS. ISS describes short children with normal GH secretion. ISS is a condition in which the height of the individual is more than 2 standard deviations below the corresponding mean height for a given age, sex and population, without evidence of systemic, endocrine, nutritional or chromosomal abnormalities. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Number of Adverse Events (AEs) Reported in Children Born Small for Gestational Age- Weeks 0 to 26 | From baseline (Week 0) to Week 26 | This outcome measure reported number of AEs in children with short stature for indication SGA. Children with SGA are born small for gestational age with insufficient catch-up growth by 2 years of age or older. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Height Velocity Reported for Idiopathic Short Stature | From Baseline (Week 0) to Week 26 | This outcome measure reported height velocity in children with short stature for indication ISS. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/ (time from baseline to 26 weeks visit in years). |
| Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Children Born Small for Gestational Age | From baseline (Week 0) to Week 26 | This outcome measure reported number of AEs possibly or probably related to somapacitan reported in children with short stature for indication SGA. Children with SGA are born small for gestational age with insufficient catch-up growth by 2 years of age or older. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Turner Syndrome | From baseline (Week 0) to Week 26 | This outcome measure reported number of AEs possibly or probably related to somapacitan reported in participants with short stature for indication TS. TS is a chromosomal disorder which leads to short stature. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Noonan Syndrome | From baseline (Week 0) to Week 26 | This outcome measure reported number of AEs possibly or probably related to somapacitan reported in participants with short stature for indication NS. An NS is a genetically heterogeneous developmental disorder characterized by postnatally reduced growth and other major disorders. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Idiopathic Short Stature | From baseline (Week 0) to Week 26 | This outcome measure reported number of AEs possibly or probably related to somapacitan in participants with short stature for indication ISS. ISS describes short children with normal GH secretion and it is a condition in which the height of the individual is more than 2 standard deviations below the corresponding mean height for a given age, sex and population, without evidence of systemic, endocrine, nutritional or chromosomal abnormalities. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Number of Adverse Events Reported Long-term Safety for Children Born Small for Gestational Age- Weeks 0 to 156 | From baseline (Week 0) to Week 156 | This outcome measure reported long-term safety in terms of number of AEs in children with short stature for indication SGA. Children with SGA are born small for gestational age with insufficient catch-up growth by 2 years of age or older. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Number of Adverse Events Reported Long-term Safety for Turner Syndrome- Weeks 0 to 156 | From baseline (Week 0) to Week 156 | This outcome measure reported long-term safety in terms of number of AEs in participants with short stature for indication TS. TS is a chromosomal disorder which leads to short stature. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Number of Adverse Events Reported Long-term Safety for Noonan Syndrome- Weeks 0 to 156 | From baseline (Week 0) to Week 156 | This outcome measure reported long-term safety in terms of number of AEs in participants with short stature for indication NS which is a genetically heterogeneous developmental disorder characterized by postnatally reduced growth and other major disorders. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Number of Adverse Events Reported Long-term Safety for Idiopathic Short Stature- Weeks 0 to 156 | From baseline (Week 0) to Week 156 | This outcome measure reported long-term safety in terms of number of AEs in participants with short stature for indication ISS. ISS describes short children with normal GH secretion. ISS is a condition in which the height of the individual is more than 2 standard deviations below the corresponding mean height for a given age, sex and population, without evidence of systemic, endocrine, nutritional or chromosomal abnormalities. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment. |
| Change in Height Standard Deviation Scores (SDS) Reported for Children Born Small for Gestational Age | Baseline (Week 0), Week 26 | This outcome measure reported height standard deviation scores in children with short stature for indication SGA. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit and SD indicates the standard deviation (SD). The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline. |
| Change in Height Standard Deviation Scores Reported for Turner Syndrome | Baseline (Week 0), Week 26 | This outcome measure reported height standard deviation scores in children with short stature for indication TS. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline. |
| Change in Height Standard Deviation Scores Reported for Noonan Syndrome | Baseline (Week 0), Week 26 | This outcome measure reported height standard deviation scores in children with short stature for indication NS. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline. |
| Change in Height Standard Deviation Scores Reported for Idiopathic Short Stature | Baseline (Week 0), Week 26 | This outcome measure reported height standard deviation scores in children with short stature for indication ISS. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline. |
| Change in Height Velocity Standard Deviation Scores Reported Separately for Children Born Small for Gestational Age | Baseline (Week 0), Week 26 | This outcome measure reported change in height velocity SDS in children with short stature for indication SGA. Change in height velocity SDS at week 26 was calculated as the height velocity SDS value at baseline Week 0 subtracted from the height velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Change in Height Velocity Standard Deviation Scores Reported Separately for Turner Syndrome | Baseline (Week 0), Week 26 | This outcome measure reported change in height velocity SDS in children with short stature for indication TS. Change in height velocity SDS at week 26 was calculated as the height velocity SDS value at baseline (Week 0) subtracted from the height velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Change in Height Velocity Standard Deviation Scores Reported for Noonan Syndrome | Baseline (Week 0), Week 26 | This outcome measure reported change in height velocity SDS in children with short stature for indication NS. Change in height velocity SDS at Week 26 was the Height Velocity SDS value at baseline (Week 0) subtracted from the Height Velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Change in Height Velocity Standard Deviation Scores Reported Separately for Idiopathic Short Stature | Baseline (Week 0), Week 26 | This outcome measure reported change in height velocity SDS in children with short stature for indication ISS. Change in height velocity SDS at Week 26 was the Height Velocity SDS value at baseline (Week 0) subtracted from the Height Velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Weeks 4, 8, 13, 20 and 26 | The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children with idiopathic short stature who were either naïve or non-naïve to GH treatment. |
| Change in Insulin-like Growth Factor 1 (IGF-1) Standard Deviation Score Reported for Children Born Small for Gestational Age | Baseline (Week 0), Week 26 | This outcome measure reported change in IGF-1 SDS in children with short stature for indication SGA. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported for Turner Syndrome | Baseline (Week 0), Week 26 | This outcome measure reported change in IGF-1 SDS in children with short stature for indication TS. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported for Noonan Syndrome | Baseline (Week 0), Week 26 | This outcome measure reported change in IGF-1 SDS in children with short stature for indication NS. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported Separately for Idiopathic Short Stature | Baseline (Week 0), Week 26 | This outcome measure reported change in IGF-1 SDS in children with short stature for indication ISS. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Change in Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) SDS Reported for Children Born Small for Gestational Age | Baseline (week 0), Week 26 | This outcome measure reported change in IGFBP-3 scores in children with short stature for indication SGA. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Turner Syndrome | Baseline (Week 0), Week 26 | This outcome measure reported change in IGFBP-3 scores in children with short stature for indication TS. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Noonan Syndrome | Baseline (Week 0), Week 26 | This outcome measure reported change in IGFBP-3 scores in children with short stature for indication NS. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Idiopathic Short Stature | Baseline (Week 0), Week 26 | This outcome measure reported change in IGFBP-3 scores in children with short stature for indication ISS. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline. |
| Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Weeks 4, 8, 13, 20 and 26 | The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children born small for gestational age who were either naïve or non-naïve to GH treatment. |
| Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Weeks 4, 8, 13, 20 and 26 | The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children with Turner Syndrome who were either naïve or non-naïve to GH treatment. |
| Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Weeks 4, 8, 13, 20 and 26 | The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children with Noonan Syndrome who were either naïve or non-naïve to GH treatment. |
| Height Velocity Reported Children Born Small for Gestational Age | From Baseline (Week 0) to Week 26 | This outcome measure reported height velocity in children with short stature for indication SGA. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/ (time from baseline to 26 weeks visit in years). |
| Height Velocity Reported for Turner Syndrome | From Baseline (Week 0) to Week 26 | This outcome measure reported height velocity in children with short stature for indication TS. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/ (time from baseline to 26 weeks visit in years). |
| Height Velocity Reported for Noonan Syndrome | From Baseline (Week 0) to Week 26 | This outcome measure reported height velocity in children with short stature for indication NS. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/(time from baseline to 26 weeks visit in years). |
Countries
Malaysia, Netherlands, Poland, South Korea, Spain, United States
Contacts
STUDY_DIRECTORClinical Transparency (dept. 2834)
Novo Nordisk A/S
Participant flow
Recruitment details
Participants were screened and assigned to treatment across 14 sites in 5 countries.
Pre-assignment details
A total of 47 participants with short stature either born small for gestational age(SGA), Noonan syndrome(NS), Turner syndrome(TS), or idiopathic short stature(ISS) were either treatment naïve or previously treated with growth hormone(GH) were enrolled & exposed to once weekly dosing of somapacitan 0.24 milligrams per kilogram per week(mg/kg/week). 26-week main phase is followed by 130-week extension phase I. Study was ongoing at data cut-off date(24Nov2024).
Participants by arm
| Arm | Count |
|---|---|
| SGA_somapacitan (GH Treatment Naïve) Participants with small for gestational age who were treatment naïve subcutaneously received a dose of 0.24 mg/kg/week somapacitan once weekly for 26-week main phase followed by an ongoing 130-week extension phase I. | 4 |
| SGA_somapacitan (Previously Treated With GH) Participants with small for gestational age who were previously treated with GH, subcutaneously received a dose of 0.24 mg/kg/week somapacitan once weekly for 26-week main phase followed by an ongoing 130-week extension phase I. | 8 |
| TS_somapacitan (GH Treatment Naïve) Participants with Turner syndrome who were treatment naïve subcutaneously received a dose of 0.24 mg/kg/week somapacitan once weekly for 26-week main phase followed by an ongoing 130-week extension phase I. | 3 |
| TS_somapacitan (Previously Treated With GH) Participants with Turner syndrome who were previously treated with GH, subcutaneously received a dose of 0.24 mg/kg/week somapacitan once weekly for 26-week main phase followed by an ongoing 130-week extension phase I. | 8 |
| NS_somapacitan (GH Treatment Naïve) Participants with Noonan syndrome who were treatment naïve subcutaneously received a dose of 0.24 mg/kg/week somapacitan once weekly for 26-week main phase followed by an ongoing 130-week extension phase I. | 6 |
| NS_somapacitan (Previously Treated With GH) Participants with Noonan syndrome who were previously treated with GH, subcutaneously received a dose of 0.24 mg/kg/week somapacitan once weekly for 26-week main phase followed by an ongoing 130-week extension phase I. | 7 |
| ISS_somapacitan (GH Treatment Naïve) Participants with idiopathic short stature who were treatment naïve subcutaneously received a dose of 0.24 mg/kg/week somapacitan once weekly for 26-week main phase followed by an ongoing 130-week extension phase I. | 2 |
| ISS_somapacitan (Previously Treated With GH) Participants with idiopathic short stature who were were previously treated with GH, subcutaneously received a dose of 0.24 mg/kg/week somapacitan once weekly for 26-week main phase followed by an ongoing 130-week extension phase I. | 9 |
| Total | 47 |
Baseline characteristics
| Characteristic | SGA_somapacitan (GH Treatment Naïve) | SGA_somapacitan (Previously Treated With GH) | TS_somapacitan (GH Treatment Naïve) | TS_somapacitan (Previously Treated With GH) | NS_somapacitan (GH Treatment Naïve) | NS_somapacitan (Previously Treated With GH) | ISS_somapacitan (GH Treatment Naïve) | ISS_somapacitan (Previously Treated With GH) | Total |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | 12.50 Years STANDARD_DEVIATION 2.08 | 11.75 Years STANDARD_DEVIATION 1.67 | 10.67 Years STANDARD_DEVIATION 1.15 | 11.00 Years STANDARD_DEVIATION 1.2 | 12.67 Years STANDARD_DEVIATION 2.07 | 12.29 Years STANDARD_DEVIATION 1.5 | 14.50 Years STANDARD_DEVIATION 0.71 | 11.44 Years STANDARD_DEVIATION 1.24 | 11.87 Years STANDARD_DEVIATION 1.67 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants | 2 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 4 Participants | 8 Participants | 3 Participants | 6 Participants | 6 Participants | 7 Participants | 2 Participants | 9 Participants | 45 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants | 1 Participants | 1 Participants | 1 Participants | 1 Participants | 1 Participants | 1 Participants | 3 Participants | 11 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 2 Participants | 7 Participants | 2 Participants | 7 Participants | 5 Participants | 6 Participants | 1 Participants | 6 Participants | 36 Participants |
| Sex: Female, Male Female | 2 Participants | 3 Participants | 3 Participants | 8 Participants | 2 Participants | 2 Participants | 0 Participants | 6 Participants | 26 Participants |
| Sex: Female, Male Male | 2 Participants | 5 Participants | 0 Participants | 0 Participants | 4 Participants | 5 Participants | 2 Participants | 3 Participants | 21 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk |
|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 4 | 0 / 8 | 0 / 3 | 0 / 8 | 0 / 6 | 0 / 7 | 0 / 2 | 0 / 9 |
| other Total, other adverse events | 3 / 4 | 5 / 8 | 2 / 3 | 7 / 8 | 4 / 6 | 6 / 7 | 2 / 2 | 8 / 9 |
| serious Total, serious adverse events | 0 / 4 | 1 / 8 | 0 / 3 | 1 / 8 | 0 / 6 | 0 / 7 | 0 / 2 | 1 / 9 |
Outcome results
Primary
Number of Adverse Events (AEs) Reported in Children Born Small for Gestational Age- Weeks 0 to 26
This outcome measure reported number of AEs in children with short stature for indication SGA. Children with SGA are born small for gestational age with insufficient catch-up growth by 2 years of age or older. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 26
Population: Safety analysis set included all participants who were exposed to study treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Number of Adverse Events (AEs) Reported in Children Born Small for Gestational Age- Weeks 0 to 26 | 4 Events |
| SGA_somapacitan (Previously Treated With GH) | Number of Adverse Events (AEs) Reported in Children Born Small for Gestational Age- Weeks 0 to 26 | 10 Events |
Primary
Number of Adverse Events Reported for Idiopathic Short Stature (ISS)- Weeks 0 to 26
This outcome measure reported number of AEs in participants with short stature for indication ISS. ISS describes short children with normal GH secretion. ISS is a condition in which the height of the individual is more than 2 standard deviations below the corresponding mean height for a given age, sex and population, without evidence of systemic, endocrine, nutritional or chromosomal abnormalities. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 26
Population: Safety analysis set included all participants who were exposed to study treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Number of Adverse Events Reported for Idiopathic Short Stature (ISS)- Weeks 0 to 26 | 9 Events |
| SGA_somapacitan (Previously Treated With GH) | Number of Adverse Events Reported for Idiopathic Short Stature (ISS)- Weeks 0 to 26 | 22 Events |
Primary
Number of Adverse Events Reported for Noonan Syndrome- Weeks 0 to 26
This outcome measure reported number of AEs in participants with short stature for indication NS which is a genetically heterogeneous developmental disorder characterized by postnatally reduced growth and other major disorders. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 26
Population: Safety analysis set included all participants who were exposed to study treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Number of Adverse Events Reported for Noonan Syndrome- Weeks 0 to 26 | 13 Events |
| SGA_somapacitan (Previously Treated With GH) | Number of Adverse Events Reported for Noonan Syndrome- Weeks 0 to 26 | 11 Events |
Primary
Number of Adverse Events Reported for Turner Syndrome (TS)- Weeks 0 to 26
This outcome measure reported number of AEs in participants with short stature for indication TS. TS is a chromosomal disorder which leads to short stature. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 26
Population: Safety analysis set included all participants who were exposed to study treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Number of Adverse Events Reported for Turner Syndrome (TS)- Weeks 0 to 26 | 4 Events |
| SGA_somapacitan (Previously Treated With GH) | Number of Adverse Events Reported for Turner Syndrome (TS)- Weeks 0 to 26 | 18 Events |
Secondary
Change in Height Standard Deviation Scores Reported for Idiopathic Short Stature
This outcome measure reported height standard deviation scores in children with short stature for indication ISS. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Height Standard Deviation Scores Reported for Idiopathic Short Stature | 0.22 standard deviation score | Standard Deviation 0.15 |
| SGA_somapacitan (Previously Treated With GH) | Change in Height Standard Deviation Scores Reported for Idiopathic Short Stature | 0.14 standard deviation score | Standard Deviation 0.18 |
Secondary
Change in Height Standard Deviation Scores Reported for Noonan Syndrome
This outcome measure reported height standard deviation scores in children with short stature for indication NS. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Height Standard Deviation Scores Reported for Noonan Syndrome | 0.35 standard deviation score | Standard Deviation 0.26 |
| SGA_somapacitan (Previously Treated With GH) | Change in Height Standard Deviation Scores Reported for Noonan Syndrome | 0.11 standard deviation score | Standard Deviation 0.14 |
Secondary
Change in Height Standard Deviation Scores Reported for Turner Syndrome
This outcome measure reported height standard deviation scores in children with short stature for indication TS. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Height Standard Deviation Scores Reported for Turner Syndrome | 0.40 standard deviation score | Standard Deviation 0.3 |
| SGA_somapacitan (Previously Treated With GH) | Change in Height Standard Deviation Scores Reported for Turner Syndrome | 0.00 standard deviation score | Standard Deviation 0.23 |
Secondary
Change in Height Standard Deviation Scores (SDS) Reported for Children Born Small for Gestational Age
This outcome measure reported height standard deviation scores in children with short stature for indication SGA. Height SDS at Week 26 was derived as the Height SDS value at baseline (Week 0) subtracted from the Height SDS value at Week 26. Height SDS is derived as: Height SDSi = ({\[Heighti/population median\]\^Skewness}-1)/(Skewness∗population SD); where i indicates the visit and SD indicates the standard deviation (SD). The population median and standard deviation are the ones corresponding to the age at visit i. The population median and standard deviation and skewness are based on reference data. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height. The positive score indicates that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Height Standard Deviation Scores (SDS) Reported for Children Born Small for Gestational Age | 0.50 standard deviation score | Standard Deviation 0.21 |
| SGA_somapacitan (Previously Treated With GH) | Change in Height Standard Deviation Scores (SDS) Reported for Children Born Small for Gestational Age | 0.24 standard deviation score | Standard Deviation 0.13 |
Secondary
Change in Height Velocity Standard Deviation Scores Reported for Noonan Syndrome
This outcome measure reported change in height velocity SDS in children with short stature for indication NS. Change in height velocity SDS at Week 26 was the Height Velocity SDS value at baseline (Week 0) subtracted from the Height Velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Height Velocity Standard Deviation Scores Reported for Noonan Syndrome | 3.43 standard deviation score | Standard Deviation 2.75 |
| SGA_somapacitan (Previously Treated With GH) | Change in Height Velocity Standard Deviation Scores Reported for Noonan Syndrome | 0.22 standard deviation score | Standard Deviation 0.93 |
Secondary
Change in Height Velocity Standard Deviation Scores Reported Separately for Children Born Small for Gestational Age
This outcome measure reported change in height velocity SDS in children with short stature for indication SGA. Change in height velocity SDS at week 26 was calculated as the height velocity SDS value at baseline Week 0 subtracted from the height velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Height Velocity Standard Deviation Scores Reported Separately for Children Born Small for Gestational Age | 3.53 standard deviation score | Standard Deviation 2.49 |
| SGA_somapacitan (Previously Treated With GH) | Change in Height Velocity Standard Deviation Scores Reported Separately for Children Born Small for Gestational Age | 1.04 standard deviation score | Standard Deviation 1.18 |
Secondary
Change in Height Velocity Standard Deviation Scores Reported Separately for Idiopathic Short Stature
This outcome measure reported change in height velocity SDS in children with short stature for indication ISS. Change in height velocity SDS at Week 26 was the Height Velocity SDS value at baseline (Week 0) subtracted from the Height Velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Height Velocity Standard Deviation Scores Reported Separately for Idiopathic Short Stature | 2.89 standard deviation score | Standard Deviation 0.85 |
| SGA_somapacitan (Previously Treated With GH) | Change in Height Velocity Standard Deviation Scores Reported Separately for Idiopathic Short Stature | -0.32 standard deviation score | Standard Deviation 1.9 |
Secondary
Change in Height Velocity Standard Deviation Scores Reported Separately for Turner Syndrome
This outcome measure reported change in height velocity SDS in children with short stature for indication TS. Change in height velocity SDS at week 26 was calculated as the height velocity SDS value at baseline (Week 0) subtracted from the height velocity SDS value at Week 26. Height Velocity SDS is derived as: HV SDSi = (HVi - population mean HV)/population SD; where i indicates the visit. The population mean and standard deviation corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater height velocity. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Height Velocity Standard Deviation Scores Reported Separately for Turner Syndrome | 3.45 standard deviation score | Standard Deviation 1.41 |
| SGA_somapacitan (Previously Treated With GH) | Change in Height Velocity Standard Deviation Scores Reported Separately for Turner Syndrome | -0.48 standard deviation score | Standard Deviation 1.84 |
Secondary
Change in Insulin-like Growth Factor 1 (IGF-1) Standard Deviation Score Reported for Children Born Small for Gestational Age
This outcome measure reported change in IGF-1 SDS in children with short stature for indication SGA. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Insulin-like Growth Factor 1 (IGF-1) Standard Deviation Score Reported for Children Born Small for Gestational Age | 1.61 standard deviation score | Standard Deviation 1.52 |
| SGA_somapacitan (Previously Treated With GH) | Change in Insulin-like Growth Factor 1 (IGF-1) Standard Deviation Score Reported for Children Born Small for Gestational Age | 1.39 standard deviation score | Standard Deviation 1.14 |
Secondary
Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported for Noonan Syndrome
This outcome measure reported change in IGF-1 SDS in children with short stature for indication NS. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported for Noonan Syndrome | 1.01 standard deviation score | Standard Deviation 1.38 |
| SGA_somapacitan (Previously Treated With GH) | Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported for Noonan Syndrome | 2.23 standard deviation score | Standard Deviation 1.87 |
Secondary
Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported for Turner Syndrome
This outcome measure reported change in IGF-1 SDS in children with short stature for indication TS. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported for Turner Syndrome | 0.88 standard deviation score | Standard Deviation 0.43 |
| SGA_somapacitan (Previously Treated With GH) | Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported for Turner Syndrome | -0.09 standard deviation score | Standard Deviation 1.52 |
Secondary
Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported Separately for Idiopathic Short Stature
This outcome measure reported change in IGF-1 SDS in children with short stature for indication ISS. Change in IGF-I SDS was derived as IGF-1 SDS value at baseline Week 0 subtracted from the IGF-I SDS value at Week 26. IGF-I SDS is derived as: IGF - I SDSi = ({\[IGF - I i\]/population median}\^Skewness - 1)/ Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGF-1. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported Separately for Idiopathic Short Stature | 2.79 standard deviation score | Standard Deviation 0.52 |
| SGA_somapacitan (Previously Treated With GH) | Change in Insulin-like Growth Factor 1 Standard Deviation Score Reported Separately for Idiopathic Short Stature | 1.66 standard deviation score | Standard Deviation 1.28 |
Secondary
Change in Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) SDS Reported for Children Born Small for Gestational Age
This outcome measure reported change in IGFBP-3 scores in children with short stature for indication SGA. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) SDS Reported for Children Born Small for Gestational Age | 0.69 standard deviation score | Standard Deviation 0.93 |
| SGA_somapacitan (Previously Treated With GH) | Change in Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) SDS Reported for Children Born Small for Gestational Age | 1.22 standard deviation score | Standard Deviation 1 |
Secondary
Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Idiopathic Short Stature
This outcome measure reported change in IGFBP-3 scores in children with short stature for indication ISS. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Idiopathic Short Stature | 1.22 standard deviation score | Standard Deviation 0.18 |
| SGA_somapacitan (Previously Treated With GH) | Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Idiopathic Short Stature | 1.08 standard deviation score | Standard Deviation 1.43 |
Secondary
Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Noonan Syndrome
This outcome measure reported change in IGFBP-3 scores in children with short stature for indication NS. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Noonan Syndrome | 0.66 standard deviation score | Standard Deviation 0.97 |
| SGA_somapacitan (Previously Treated With GH) | Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Noonan Syndrome | 0.67 standard deviation score | Standard Deviation 1.08 |
Secondary
Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Turner Syndrome
This outcome measure reported change in IGFBP-3 scores in children with short stature for indication TS. Change in IGFBP-3 SCS at Week 26 was derived as the IGFBP-3 SDS value at baseline Week 0 subtracted from IGFBP-3 SDS value at Week 26. IGFBP-3 SDS is derived as: IGFBP - 3 SDSi = ({\[IGFBP - 3 i/population median\]\^Skewness} - 1)/Skewness ∗ population SD; where i indicates the visit. The population median and standard deviation are the ones corresponding to the age at visit i. The score ranges from -10 (minimum) to +10 (maximum), where the greater value indicated greater IGFBP-3. The positive score indicated that the value is closer to or above the reference population compared to baseline.
Time frame: Baseline (Week 0), Week 26
Population: Full analysis set included all participants assigned to study intervention. Here 'Number of participants analysed' signifies number of participants with available data for particular timepoint.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Turner Syndrome | 0.39 standard deviation score | Standard Deviation 0.48 |
| SGA_somapacitan (Previously Treated With GH) | Change in Insulin-like Growth Factor Binding Protein-3 SDS Reported for Turner Syndrome | 0.05 standard deviation score | Standard Deviation 1.45 |
Secondary
Height Velocity Reported Children Born Small for Gestational Age
This outcome measure reported height velocity in children with short stature for indication SGA. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/ (time from baseline to 26 weeks visit in years).
Time frame: From Baseline (Week 0) to Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Height Velocity Reported Children Born Small for Gestational Age | 11.3 centimeters per year (cm/year) | Standard Deviation 3.8 |
| SGA_somapacitan (Previously Treated With GH) | Height Velocity Reported Children Born Small for Gestational Age | 9.5 centimeters per year (cm/year) | Standard Deviation 2 |
Secondary
Height Velocity Reported for Idiopathic Short Stature
This outcome measure reported height velocity in children with short stature for indication ISS. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/ (time from baseline to 26 weeks visit in years).
Time frame: From Baseline (Week 0) to Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Height Velocity Reported for Idiopathic Short Stature | 9.2 cm/year | Standard Deviation 3.5 |
| SGA_somapacitan (Previously Treated With GH) | Height Velocity Reported for Idiopathic Short Stature | 7.9 cm/year | Standard Deviation 2.4 |
Secondary
Height Velocity Reported for Noonan Syndrome
This outcome measure reported height velocity in children with short stature for indication NS. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/(time from baseline to 26 weeks visit in years).
Time frame: From Baseline (Week 0) to Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Height Velocity Reported for Noonan Syndrome | 9.1 cm/year | Standard Deviation 2.3 |
| SGA_somapacitan (Previously Treated With GH) | Height Velocity Reported for Noonan Syndrome | 6.4 cm/year | Standard Deviation 2 |
Secondary
Height Velocity Reported for Turner Syndrome
This outcome measure reported height velocity in children with short stature for indication TS. Height velocity at week 26 was derived as: (height at 26 weeks visit - height at baseline)/ (time from baseline to 26 weeks visit in years).
Time frame: From Baseline (Week 0) to Week 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Height Velocity Reported for Turner Syndrome | 10.2 cm/year | Standard Deviation 2.2 |
| SGA_somapacitan (Previously Treated With GH) | Height Velocity Reported for Turner Syndrome | 5.8 cm/year | Standard Deviation 2 |
Secondary
Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Children Born Small for Gestational Age
This outcome measure reported number of AEs possibly or probably related to somapacitan reported in children with short stature for indication SGA. Children with SGA are born small for gestational age with insufficient catch-up growth by 2 years of age or older. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 26
Population: Safety analysis set included all participants who were exposed to study treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Children Born Small for Gestational Age | 1 Events |
| SGA_somapacitan (Previously Treated With GH) | Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Children Born Small for Gestational Age | 1 Events |
Secondary
Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Idiopathic Short Stature
This outcome measure reported number of AEs possibly or probably related to somapacitan in participants with short stature for indication ISS. ISS describes short children with normal GH secretion and it is a condition in which the height of the individual is more than 2 standard deviations below the corresponding mean height for a given age, sex and population, without evidence of systemic, endocrine, nutritional or chromosomal abnormalities. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 26
Population: Safety analysis set included all participants who were exposed to study treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Idiopathic Short Stature | 0 Events |
| SGA_somapacitan (Previously Treated With GH) | Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Idiopathic Short Stature | 1 Events |
Secondary
Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Noonan Syndrome
This outcome measure reported number of AEs possibly or probably related to somapacitan reported in participants with short stature for indication NS. An NS is a genetically heterogeneous developmental disorder characterized by postnatally reduced growth and other major disorders. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 26
Population: Safety analysis set included all participants who were exposed to study treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Noonan Syndrome | 3 Events |
| SGA_somapacitan (Previously Treated With GH) | Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Noonan Syndrome | 2 Events |
Secondary
Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Turner Syndrome
This outcome measure reported number of AEs possibly or probably related to somapacitan reported in participants with short stature for indication TS. TS is a chromosomal disorder which leads to short stature. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 26
Population: Safety analysis set included all participants who were exposed to study treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Turner Syndrome | 0 Events |
| SGA_somapacitan (Previously Treated With GH) | Number of Adverse Events Possibly or Probably Related to Somapacitan Reported for Turner Syndrome | 0 Events |
Secondary
Number of Adverse Events Reported Long-term Safety for Children Born Small for Gestational Age- Weeks 0 to 156
This outcome measure reported long-term safety in terms of number of AEs in children with short stature for indication SGA. Children with SGA are born small for gestational age with insufficient catch-up growth by 2 years of age or older. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 156
Secondary
Number of Adverse Events Reported Long-term Safety for Idiopathic Short Stature- Weeks 0 to 156
This outcome measure reported long-term safety in terms of number of AEs in participants with short stature for indication ISS. ISS describes short children with normal GH secretion. ISS is a condition in which the height of the individual is more than 2 standard deviations below the corresponding mean height for a given age, sex and population, without evidence of systemic, endocrine, nutritional or chromosomal abnormalities. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 156
Secondary
Number of Adverse Events Reported Long-term Safety for Noonan Syndrome- Weeks 0 to 156
This outcome measure reported long-term safety in terms of number of AEs in participants with short stature for indication NS which is a genetically heterogeneous developmental disorder characterized by postnatally reduced growth and other major disorders. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 156
Secondary
Number of Adverse Events Reported Long-term Safety for Turner Syndrome- Weeks 0 to 156
This outcome measure reported long-term safety in terms of number of AEs in participants with short stature for indication TS. TS is a chromosomal disorder which leads to short stature. An AE is any untoward medical occurrence in a clinical study participant that is temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of an study treatment.
Time frame: From baseline (Week 0) to Week 156
Secondary
Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age
The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children born small for gestational age who were either naïve or non-naïve to GH treatment.
Time frame: Weeks 4, 8, 13, 20 and 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Week 8 | 1.800 nanograms per milliliter (ng/mL) | Standard Deviation 0.857 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Week 20 | 2.345 nanograms per milliliter (ng/mL) | Standard Deviation 1.331 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Week 13 | 62.280 nanograms per milliliter (ng/mL) | Standard Deviation 92.161 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Week 26 | 5.928 nanograms per milliliter (ng/mL) | Standard Deviation 4.904 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Week 4 | 518.948 nanograms per milliliter (ng/mL) | Standard Deviation 58.993 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Week 26 | 8.364 nanograms per milliliter (ng/mL) | Standard Deviation 5.075 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Week 4 | 339.226 nanograms per milliliter (ng/mL) | Standard Deviation 205.265 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Week 8 | 2.204 nanograms per milliliter (ng/mL) | Standard Deviation 0.556 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Week 13 | 78.530 nanograms per milliliter (ng/mL) | Standard Deviation 79.022 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Children Born Small for Gestational Age | Week 20 | 4.123 nanograms per milliliter (ng/mL) | Standard Deviation 3.263 |
Secondary
Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature
The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children with idiopathic short stature who were either naïve or non-naïve to GH treatment.
Time frame: Weeks 4, 8, 13, 20 and 26
Population: Full analysis set included all participants assigned to study intervention. Here, 'Number Analyzed' signifies number of participants with available data for particular timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Week 8 | 9.535 nanograms per milliliter (ng/mL) | Standard Deviation 0.94 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Week 20 | 7.070 nanograms per milliliter (ng/mL) | Standard Deviation 3.564 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Week 13 | 403.525 nanograms per milliliter (ng/mL) | Standard Deviation 156.235 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Week 26 | 16.630 nanograms per milliliter (ng/mL) | — |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Week 4 | 344.415 nanograms per milliliter (ng/mL) | Standard Deviation 313.128 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Week 26 | 16.461 nanograms per milliliter (ng/mL) | Standard Deviation 15.509 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Week 4 | 401.444 nanograms per milliliter (ng/mL) | Standard Deviation 178.512 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Week 8 | 57.152 nanograms per milliliter (ng/mL) | Standard Deviation 145.426 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Week 13 | 74.361 nanograms per milliliter (ng/mL) | Standard Deviation 65.211 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Idiopathic Short Stature | Week 20 | 4.876 nanograms per milliliter (ng/mL) | Standard Deviation 4.495 |
Secondary
Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome
The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children with Noonan Syndrome who were either naïve or non-naïve to GH treatment.
Time frame: Weeks 4, 8, 13, 20 and 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Week 8 | 3.102 nanograms per milliliter (ng/mL) | Standard Deviation 2.197 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Week 20 | 34.043 nanograms per milliliter (ng/mL) | Standard Deviation 69.759 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Week 13 | 102.647 nanograms per milliliter (ng/mL) | Standard Deviation 164.113 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Week 26 | 13.057 nanograms per milliliter (ng/mL) | Standard Deviation 22.222 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Week 4 | 249.278 nanograms per milliliter (ng/mL) | Standard Deviation 225.237 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Week 26 | 42.834 nanograms per milliliter (ng/mL) | Standard Deviation 58.984 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Week 4 | 254.626 nanograms per milliliter (ng/mL) | Standard Deviation 175.518 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Week 8 | 31.343 nanograms per milliliter (ng/mL) | Standard Deviation 58.845 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Week 13 | 134.501 nanograms per milliliter (ng/mL) | Standard Deviation 188.934 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Noonan Syndrome | Week 20 | 3.869 nanograms per milliliter (ng/mL) | Standard Deviation 2.376 |
Secondary
Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome
The steady state pharmacokinetics in terms of Cavg was evaluated for once-weekly somapacitan in children with Turner Syndrome who were either naïve or non-naïve to GH treatment.
Time frame: Weeks 4, 8, 13, 20 and 26
Population: Full analysis set included all participants assigned to study intervention.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Week 8 | 2.187 nanograms per milliliter (ng/mL) | Standard Deviation 0.335 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Week 20 | 2.487 nanograms per milliliter (ng/mL) | Standard Deviation 1.232 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Week 13 | 75.400 nanograms per milliliter (ng/mL) | Standard Deviation 125.123 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Week 26 | 4.363 nanograms per milliliter (ng/mL) | Standard Deviation 2.89 |
| SGA_somapacitan (GH Treatment Naïve) | Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Week 4 | 287.777 nanograms per milliliter (ng/mL) | Standard Deviation 191.086 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Week 26 | 8.574 nanograms per milliliter (ng/mL) | Standard Deviation 5.304 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Week 4 | 384.130 nanograms per milliliter (ng/mL) | Standard Deviation 252.372 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Week 8 | 3.285 nanograms per milliliter (ng/mL) | Standard Deviation 2.018 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Week 13 | 93.753 nanograms per milliliter (ng/mL) | Standard Deviation 162.274 |
| SGA_somapacitan (Previously Treated With GH) | Weekly Average Somapacitan Concentration (Cavg) Reported for Turner Syndrome | Week 20 | 2.871 nanograms per milliliter (ng/mL) | Standard Deviation 1.018 |